BlueRock announces first patient receives investigational therapy in CLARICO phase 1/2a trial

Overview

Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, announced that the first patient received the investigational therapy in CLARICO, a phase 1/2a clinical trial of OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for treating primary photoreceptor (PR) diseases, which are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy.

Statement from Amit Rakhit: BlueRock’s CD&MO

The initiation of the CLARICO trial represents a key milestone for the OpCT-001 programme,” said Amit Rakhit, MD, MBA, BlueRock’s chief development and medical officer. “We believe OpCT-001 holds significant promise as a novel therapeutic approach for restoring vision in people living with primary photoreceptor diseases, and we look forward to assessing its safety and tolerability profile as we advance this important programme in our pipeline.”

Phase 1/2a CLARICO Trial of OpCT-001 for Primary Photoreceptor Disease

CLARICO is a phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and includes a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.

Statement from Christian Rommel: Christian Rommel

We aim to transform treatment options for patients facing irreversible vision loss. OpCT-001, our investigational iPSC-derived cell therapy, has the potential to restore vision for individuals with primary photoreceptor disease,” said Christian Rommel, executive vice president and global head of research and development of the pharmaceuticals division at Bayer. 
“We are excited to announce the first patient in the CLARICO trial, the first-ever clinical trial for an iPSC-derived treatment in this field.”
The Primary photoreceptor diseases
Primary photoreceptor diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. OpCT-001 aims to restore vision in patients with primary photoreceptor diseases by replacing degenerated cells in the retina with functional cells. Limited treatment options currently exist for treating primary photoreceptor diseases which affect an estimated 110,000 people in the US.

OpCT-001 therapy

OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

About CLARICO trial

• CLARICO is a phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. 
• Phase 1 will focus on safety and features a dose-escalation design. phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups. 
• Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). Dose escalation in phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 to 24 legally blind participants (~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants in 2 cohorts (Cohorts 5 and 6) to evaluate 2 dose levels of OpCT-001 that will be selected based on phase 1 safety and tolerability data. 
• Phase 2 participants will be randomized 1:1 to either Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments. 

About both companies

• •BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. 
• Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition.

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